by Peter Ciszewski | Apr 9, 2018
Ellen Ritchie, MD, Associate Professor of Clinical Medicine, Weill Cornell Medical College, discusses the available treatment options for Myeloproliferative Neoplasms (MPNs). MPNs are rare, chronic blood cancers. Chronic conditions last for a long period of time and...
by Peter Ciszewski | Apr 9, 2018
Other Names: Osteitis Deformans Paget’s disease of bone is a condition involving cellular remodeling and deformity of one or more bones. The affected bones show signs of dysregulated bone remodeling at the microscopic level, specifically excessive bone breakdown...
by Peter Ciszewski | Apr 8, 2018
Glyn Edwards of Summit Therapeutics discusses endpoints of Duchenne muscular dystrophy (DMD) clinical trials. DMD is one of the most common, fatal genetic disorders diagnosed in children around the world. DMD is an X-linked disease, meaning that it predominantly...
by Peter Ciszewski | Apr 8, 2018
Luca Santarelli, MD of Therachon discusses diagnosing Achondroplasia. Therachon’s lead pipeline candidate, TA-46, a novel protein therapy in development for achondroplasia, the most common form of short-limbed dwarfism. Achondroplasia is an autosomal dominant...
by Peter Ciszewski | Apr 8, 2018
Melissa P. Wasserstein, MD, Chief of Pediatric Genetic Medicine at the Children’s Hospital at Montefiore, New York City discusses the challenges of diagnosing rare diseases. She uses Krabbe disease, Nieman-Pick disease, and 22q11.2 deletion syndrome as examples....
